Thursday, August 25, 2011

What the Rest of 2011 Holds for Celsion

Amidst a volatile US market/economic backdrop, anticipation continues to build for Celsion, particularly leading into the announcement of confirmation of at least 190 progression-free survival (PFS) events from the HEAT study to formally trigger initiation of the interim analysis. Volume has fallen significantly, and the stock has made a modest recovery back over $3, but there remain a lot of potential catalysts between now and the end of the year. As many of you probably already know, I am holding my shares for the long-haul, and do not plan on trading until at least final results of the HEAT study are out, if not, all the way through FDA approval. With that said, investors might see some opportunities surrounding these anticipated 2011 events. I've made an effort to list them chronologically based on my best guess of when they would occur.
  1. PR confirming at least 190 PFS events from HEAT trial --> triggers interim analysis
    • My guess is this happens very soon, by end of August or early September.
  2. Enrollment/treatment of first patient in the Thermodox Phase II colorectal liver mets study (CRLM)
  3. Update on the recurrent chest wall breast cancer DIGNITY Phase II study protocol
    • In my opinion, a decision to move Thermodox as earlier line therapy, which the company has clearly alluded to, could be pretty significant.
  4. Initiation of the Thermodox Phase II prostate cancer bone metastases study with partner, Philips, using MRI-guided High Intensity Focused Ultrasound (MRI-HIFU)
    • I recall the company mentioning, along with Philips, they have another meeting with the FDA in September to submit some additional data. Note that Philips' Sonalleve MRI-HIFU device is not yet approved in the US, so that will probably have its own 510k/PMA device application as well, though they are collectively being regulated as a drug-device combination.  
  5. The HEAT study interim analysis results, likely due 8 weeks after the 190+ PFS announcement. (my guess, by end of October, early November at the very latest). 
    • This is obviously the catalyst many investors are thinking about, and rightfully so. At this point, with a chance for futility being "de minimis" to quote the CEO, I think we can safely assume we are looking at either a continuation to 380 events, or overwhelming efficacy sufficient to justify an early NDA. While the study was designed for the former and that is clearly the likely outcome, speculation is all over the place. I personally give an overwhelming efficacy outcome a likelihood of 20%. If the study continues to 380 events, in all likelihood, top-line results would be out 1 year from now. As another reminder, there will not be actual data revealed if the DMC recommends a continuation to 380 events, so investors need to keep that in mind.
  6. Update on the company's carboplatin preclinical research, which, is being spearheaded by the inventor of the company's LTSL platform technology, Duke University's Dr. David Needham.
  7. The company also alluded to finally revealing the partner and product behind the "secret" fourth product in the company's pipeline (Thermodox, Docetaxel, Carboplatin being the first three). I have my guesses what this fourth product can be, but I will save that for another post ;-)
  8. Completion of a second license deal (the first being Yakult in Japan, see my Japan article) with a major big pharma company. 
    • Similar to the HEAT trial enrollment which has lagged behind company estimates for some time, so has the elusive second deal. As a reminder, the company is 100% committed to licensing Thermodox out to a major pharma partner for ex-US geographies, but Celsion is likely considering "going it alone" in the US based on prior statements. Either way, the partner, deal terms, affected geographies, etc., could be game changing for the company. As a reminder, when asked during a cc why this second deal has taken so long, the CEO stated that they are confident a deal will be made following the interim analysis. Outside of a trial halt for early efficacy, a second deal with good terms is probably the biggest catalyst for the company. A fellow poster on the yahoo mb ("Celsiodaat") and knowledgeable investor has insisted for a very long time now the potential partner could be Merck. I happen to strongly agree with that pick for a variety of reasons, not the least of which includes the fact that up until 2010, Merck had international rights to J&J's non-heat sensitive liposomal doxorubicin (Doxil, marketed outside the US in most countries as Caelyx). Between J&J and Merck, Doxil was/is $600-$700M drug. 
  9. Tying into a second deal, we should also be hearing about CMC updates for Thermodox as the company gears up for registrational batch manufacturing. 
  10. Official guidance from the European Medicines Agency (EMA) regarding Thermodox' filing strategy for the EU.
  11. Initiation of rolling NDA for Thermodox.
Yep, there are a lot of things happening outside of just the interim analysis. As a shareholder, I am quite excited at what lies ahead. As always, feel free to leave comments or ask any questions. 


1 comment:

  1. Excellent compilation of Due Diligence. Thank you for this.

    A coup1e questions I have that perhaps you can help me with:

    1. How experienced is management at getting an NDA approved? The management team is very experienced in therapeutics, but has any of them been responsible for taking a new product through to an NDA before?

    2. What does the competitive landscape look like for Thermodox? Are there any other promising products for the same indications racing to get approved that may make Thermodox moot? (Like what happened to NEOP last July, when their investors got blind-sided by a private company.)

    3. How significant and rare is the "Rolling NDA" process?